Onasemnogene abeparvovec for the treatment of spinal muscular atrophy

Author:

McMillan Hugh J.1ORCID,Proud Crystal M.2,Farrar Michelle A.34,Alexander Ian E.56,Muntoni Francesco78,Servais Laurent910ORCID

Affiliation:

1. Departments of Pediatrics, Neurology & Neurosurgery, Montreal Children’s Hospital, McGill University Health Centre, Montreal, Canada

2. Children’s Hospital of the King’s Daughters, Norfolk, VA, United States

3. School of Clinical Medicine, UNSW Medicine and Health, UNSW Sydney, Sydney, NSW, Australia

4. Department of Neurology, Sydney Children’s Hospital Network, Sydney, NSW, Australia

5. Gene Therapy Research Unit, Children’s Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children’s Hospitals Network, Westmead, NSW, Australia

6. Discipline of Child and Adolescent Health, Sydney Medical School, Faculty of Medicine and Health, The University of Sydney, Westmead, NSW, Australia

7. The Dubowitz Neuromuscular Centre, University College London, Great Ormond Street Institute of Child Health London, UK

8. NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London, & Great Ormond Street Hospital Trust, London, UK

9. Department of Pediatrics, Centre Hospitalier Universitaire de Liège & Université de Liège, Liège, Belgium

10. MDUK Oxford Neuromuscular Centre, University of Oxford, Oxford, UK

Funder

Novartis Gene Therapies, Inc

Publisher

Informa UK Limited

Subject

Clinical Biochemistry,Drug Discovery,Pharmacology

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